Among the proposed provisions is a change that would allow a drug to be approved with a “breakthrough therapy designation,” based on evidence from its early-stage testing. Another provision would allow manufacturers to get accelerated approval for a drug based on its effect on a surrogate endpoint—a lab test result like lowered cholesterol that predicts health benefits, but hasn't yet been shown in clinical trials to improve patient outcomes.
Given the prospect of greater scientific uncertainty, critics wonder whether Califf will remain impartial and hold the line on safety and efficacy standards. They cite his long-standing financial ties with drug companies while directing the Duke Clinical Research Institute. “The attitudes he's formed will lead him to make decisions that weigh in favor of industry instead of public health,” charged Dr. Michael Carome, director of the Health Research Group for the advocacy organization Public Citizen.
Critics of some of the reform measures proposed by Congress, including former FDA Commissioner Dr. Margaret Hamburg and her former top deputies, are more concerned about the agency's ability to simply keep up with changed regulatory standards. The agency will need additional resources to train existing and new personnel to keep up with evolving regulatory science if it is going to play its traditional role of guiding the industry through the process of developing new drugs and devices without causing unexpected harm.
“It's important for people to understand the FDA is not an obstacle to progress but really can help progress come about,” said Dr. Joshua Sharfstein, an associate dean at the Johns Hopkins Bloomberg School of Public Health and a former deputy FDA commissioner. “FDA is sometimes the agency people love to hate—but usually that comes out of a place of not understanding.”
Given the election year uncertainty, Califf will probably use the next six months to chart future plans. The one exception is an initiative on opioid abuse, which has become a major issue on the campaign trail. With the Obama administration ending in less than a year, experts say Califf will move cautiously on other issues, in hopes of maximizing his chances of being retained by the next administration—by whoever wins the election.
“I would be surprised if there were many groundbreaking positions that could be controversial politically,” said Marc Scheineson, a partner at the Washington, D.C.-based law firm Alston and Bird, who previously served as associate commissioner for legislative affairs at the FDA.